Therapeutic compositions and methods of using same

a technology of compositions and compositions, applied in the field of therapeutic compositions, can solve the problems of rapid clearance of rnai reagents (i.e., dsrna oligo) from the circulation of subjects

Inactive Publication Date: 2006-06-08
BRISTOL MYERS SQUIBB CO
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

A current challenge associated with the use of RNAi methodology in therapeutic applications is the rapid clearance of the RNAi reagent (i.e., the dsRNA oligo) from the circulation of a subject.
To date, this and other hurdles has led to the effective application of RNAi technology in only limited circumstances, such as the treatment of macular degeneration, in which the reagent is not introduced into the circulation of the subject but is injected directly into the eye of a subject.

Method used

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  • Therapeutic compositions and methods of using same
  • Therapeutic compositions and methods of using same
  • Therapeutic compositions and methods of using same

Examples

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Effect test

example 1

Treatment of a Condition

[0083] A composition is prepared that can be employed in the treatment of a condition in which it is desired to down-regulate or silence the expression or activity of a gene implicated in the condition. In one example, the condition is the presence or growth of a tumor. In another example, the condition is obesity.

[0084] A composition comprising a vasculature translocation peptide comprising the amino acid sequence CKGGRAKDC (SEQ ID NO:1), and a nucleic acid targeted agent comprising an anti-VEGFR or anti-VEGF siRNA oligo, is prepared using appropriate chemical reactions. The composition can optionally comprise a linker and a detectable label.

[0085] The composition is administered to a subject in the vicinity of a site known or suspected to be undergoing vascularization, for example by injection, or by employing an osmotic minipump.

[0086] The effect of the composition on obesity, or tumor growth and / or angiogenesis, in the site of administration can be as...

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Abstract

A targeted composition comprising a modulation molecule, such as an siRNA oligo or a small molecule, and a translocation peptide is disclosed. Methods of making and using the composition are also disclosed. The disclosed composition can be employed in a variety of therapeutic and research applications, in which it is desirable to deliver a modulation molecule, such as an siRNA oligo or a small molecule, to a particular site, such as a site of vascularization. In one aspect of a composition of the invention, a translocation peptide directs the modulation molecule to a selected location, and a modulation molecule modulates the expression or activity of a selected target disposed at or near the selected location.

Description

CROSS-REFERENCE TO RELATED APPLICATIONS [0001] This patent application claims benefit to provisional application U.S. Ser. No. 60 / 632,864 filed Dec. 3, 2004. The entire teaching of the referenced application is incorporated herein by reference.FIELD OF THE INVENTION [0002] The present invention relates generally to compositions and methods for treating a condition by modulating the activity of a selected target, or an activity mediated by a selected target. In various aspects, the invention also relates to siRNA-mediated therapeutic treatment regimes and compositions for use in such regimes. The present invention also relates to target validation methods. The compositions of the present invention comprise a modulation molecule and a translocation peptide. BACKGROUND OF THE INVENTION [0003] Since its discovery, there has been a great deal of interest in the field of RNA interference (“RNAi”). The field has been reported to hold great therapeutic potential. RNA interference generally ...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): A61K48/00A61K38/17A61K38/18
CPCA61K38/17
Inventor HO, SIEW
Owner BRISTOL MYERS SQUIBB CO
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